Stem cells therapy in amyotrophic lateral sclerosis treatment. A critical view;Trasplante de células troncales como terapia para la esclerosis lateral amiotrófca. una mirada crítica

Revista de Neurologia

Volumn 52, Issue 7, 2011, Pages 426-434

  Soler, Bernardita ^a   Fadic, Ricardo ^a   von Bernhardi, Rommy ^a  

^a Pontifcia Universidad Católica de Chile   (Spain)

Author keywords

ALS; Immunomodulation; Mesenchymal cells; Neurodegenerative disease neuroinfammation. regenerative therapy

Indexed keywords

NEUROTROPHIC FACTOR;

AMYOTROPHIC LATERAL SCLEROSIS; ARTICLE; CELL DIFFERENTIATION; CENTRAL NERVOUS SYSTEM; DISEASE COURSE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; IMMUNOMODULATION; INFLAMMATION; STEM CELL TRANSPLANTATION; ANIMAL; BIOLOGICAL THERAPY; CLINICAL TRIAL (TOPIC); CYTOLOGY; MESENCHYMAL STEM CELL; METHODOLOGY; PATHOLOGY; PATHOPHYSIOLOGY; PHYSIOLOGY; REVIEW; TREATMENT OUTCOME;

AMYOTROPHIC LATERAL SCLEROSIS; ANIMALS; CLINICAL TRIALS AS TOPIC; DISEASE PROGRESSION; HUMANS; MESENCHYMAL STEM CELLS; STEM CELL TRANSPLANTATION; TISSUE THERAPY; TREATMENT OUTCOME;

EID: 79955535805     PISSN: 02100010     EISSN: None     Source Type: Journal    
DOI: 10.33588/rn.5207.2010483     Document Type: Article

References (78)

1. Matías-Guiu J, Barcia JA, García-Verdugo JM, Galán L, Vela A, García-Ramos R. Terapia celular en la esclerosis lateral amiotrófca. Neurologia 2008; 23: 226-237.
2. Boillée S, Van de Velde CH, Cleveland D. ALS: a disease of motor neurons and their nonneuronal neighbors. Neuron 2006; 52: 39-59.
3. Pasinelli P, Brown R. Molecular biology of amyotrophic lateral sclerosis: insights from genetics. Nat Rev Neurosci 2006; 7: 710-723.
4. Goodall EF, Morrison KE. Amyotrophic lateral sclerosis (motor neuron disease): proposed mechanisms and pathways to treatment. Expert Rev Mol Med 2006; 8: 1-22.
5. Rothstein JD. Current hypotheses for the underlying biology of amyotrophic lateral sclerosis. Ann Neurol 2009; 65 (Suppl 1): S3-9.
6. Barber SC, Shaw PJ. Oxidative stress in ALS: key role in motor neuron injury and therapeutic target. Free Rad Biol 2010; 48: 629-641.
7. Sargsyan SA, Monk PN, Shaw PJ. Microglia as potential contributors to motor neuron injury in amyotrophic lateral sclerosis. Glia 2005; 51: 241-253.
8. Beers DR, Henkel JS, Xiao Q, Zhao W, Wang J, Yen AA, et al. Wild-type microglia extend survival in PU.1 knockout mice with familial amyotrophic lateral sclerosis. Proc Natl Acad Sci U SA 2006; 103: 6.
9. Marchetto MC, Muotri AR, Mu Y, Smith AM, Cezar GG, Gage FH. Non-cell-autonomous effect of human SOD1 G37R astrocytes on motor neurons derived from human embryonic stem cells. Cell Stem Cell 2008; 3: 649-657.
10. Di Giorgio FP, Boulting GL, Bobrowicz S, Eggan KC. Human embryonic stem cell-derived motor neurons are sensitive to the toxic effect of glial cells carrying an ALS-causing mutation. Cell Stem Cell 2008; 3: 637-648.
11. Yamanaka K, Chun SJ, Boillee S, Fujimori-Tonou N, Yamashita H, Gutmann DH, et al. Astrocytes as determinants of disease progression in inherited amyotrophic lateral sclerosis. Nat Neurosci 2008; 11: 251-253.
12. Nagai M, Re DB, Nagata T, Chalazonitis A, Jessell TM, Wichterle H, et al. Astrocytes expressing ALS-linked mutated SOD1 release factors selectively toxic to motor neurons. Nat Neurosci 2007; 10: 615-622.
13. Grosskreutz J, Van den Bosch L, Keller BU. Calcium dysregulation in amyotrophic lateral sclerosis. Cell Calcium 2010; 47: 165-174.
14. Portero A, Orive G, Hernández RM, Pedraz JL. Encapsulación de células para el tratamiento de enfermedades del sistema nervioso central. Rev Neurol 2010; 50: 409-419.
15. Siciliano G, Carlesi C, Pasquali L, Piazza S, Pietracupa S, Fornai F, et al. Clinical trials for neuroprotection in ALS. CNS Neurol Disord Drug Targets 2010; 9: 305-313.
16. Meininger V, Lacomblez L, Salacas F. What has changed with riluzole? J Neurol 2000; 247: 1918-1922.
17. Turner MR, Parton MJ, Leigh PN. Clinical trials in ALS: an overview. Semin Neurol 2001; 21: 167-175.
18. Mazzini L, Vercelli A, Ferrero I, Mareschi K, Boido M, Servo S, et al. Stem cells in amyotrophic lateral sclerosis: state of the art. Expert Opin Biol Ter 2009; 9: 1245-58.
19. Ramalho-Santos M, Willenbring H. On the origin of the term 'stem cell'. Cell Stem Cell 2007; 7: 35-38.
20. Mejía-Toiber J, Castillo CG, Giordano M. Terapia celular y terapia génica ex vivo: avances en el tratamiento de enfermedades del sistema nervioso central. Rev Neurol 2009; 49: 483-489.
21. Madhavan L, Ourednik V, Ourednik J. Neural stem/progenitor cells initiate the formation of cellular networks that provide neuroprotection by growth factor-modulated antioxidant expression. Stem Cells 2008; 26: 254-265.
22. Hyun I. Allowing innovative stem cell-based therapies outside of clinical trials: ethical and policy challenges. J Law Med Ethics 2010; 38: 277-285.
23. Houssin D. Medical use, current and future, of human stem cells. Bull Acad Natl Med 2000; 184: 1191-1198.
24. Knoepfer P. Embryonic stem cells/induced pluripotent stem cells. Stem Cells 2009; 27: 1050-1056.
25. Jiang Y, Jahagirdar BN, Reinhardt RL, Schwartz RE, Keene CD, Ortiz-González XR, et al. Pluripotency of mesenchymal stem cells derived from adult marrow. Nature 2002; 418: 41-49.
26. Woodbury D, Schwarz EJ, Prockop DJ, Black IB. Adult rat and human bone marrow stromal cells differentiate into neurons. J Neurosci Res 2000; 61: 364-370.
27. Deng W, Obrocka M, Fischer I, Prockop DJ. In vitro differentiation of human marrow stromal cells into early progenitors of neural cells by conditions that increase intracellular cyclic AMP. Biochem Biophys Res Commun 2001; 282: 148-152.
28. Kohyama J, Abe H, Shimazaki T, Koizumi A, Nakashima K, Gojo S, et al. Brain from bone: efficient metadifferentiation of marrow stroma-derived mature osteoblasts to neurons with Noggin or a demethylating agent. Differentiation 2001; 68: 235-244.
29. Kim BJ, Seo JH, Bubien JK, Oh YS. Differentiation of adult bone marrow stem cells into neuroprogenitor cells in vitro. Neuroreport 2002; 13: 1185-1188.
30. Alexanian AR, Crowe MJ, Kurpad SN. Efcient differentiation and integration of lineage-restricted neural precursors in the traumatically injured adult cat spinal cord. J Neurosci Met 2006; 150: 41-46.
31. Mareschi K, Novara M, Rustichelli D, Ferrero I, Guido D, Carbone E, et al. Neural differentiation of human mesenchymal stem cells: evidence for expression of neural markers and eag K+ channel types. Exp Hematol 2006; 34: 1563-1572.
32. Blondheim NR, Levy YS, Ben-Zur T, Burshtein A, Cherlow T, Kan I, et al. Human mesenchymal stem cells express neural genes, suggesting a neural predisposition. Stem Cells Dev 2006; 15: 141-164.
33. Terada N, Hamazaki T, Oka M, Hoki M, Mastalerz DM, Nakano Y, et al. Bone marrow cells adopt the phenotype of other cells by spontaneous cell fusion. Nature 2002; 416: 542-545.
34. Brazelton TR, Rossi FM, Keshet GI, Blau HM. From marrow to brain: expression of neuronal phenotypes in adult mice. Science 2000; 290: 1775-1779.
35. Papadeas S, Maragakis N. Advances in stem cell research for amyotrophic lateral sclerosis. Curr Opin Biotechnol 2009; 20: 545-555.
36. Clement AM, Nguyen MD, Roberts EA, Garcia ML, Boillée S, Rule M, et al. Wild-type nonneuronal cells extend survival of SOD1 mutant motor neurons in ALS mice. Science 2003; 302: 113-117.
37. Crigler L, Robey RC, Asawachaicharn A, Gaupp D, Phinney DG. Human mesenchymal stem cell subpopulations express a variety of neuro-regulatory molecules and promote neuronal cell survival and neuritogenesis. Exp Neurol 2006; 198: 54-64.
38. Vercelli A, Mereuta OM, Garbossa D, Muraca G, Mareschi K, Rustichelli D, et al. Human mesenchymal stem cell transplantation extends survival, improves motor performance and decreases neuroinflamation in mouse model of amyotrophic lateral sclerosis. Neurobiol Dis 2008; 31: 395-405.
39. Ende N, Weinstein F, Chen R, Ende M. Human umbilical cord blood effect on sod mice (amyotrophic lateral sclerosis). Life Sci 2000; 67: 53-59.
40. Willing AE, Garbuzova-Davis S, Saporta S, Mlikem M, Cahill DW, Sanberg PR. hNT neurons delay onset of motor defcits in a model of amyotrophic lateral sclerosis. Brain Res Bull 2001; 56: 525-530.
41. Garbuzova-Davis S, Willing A, Milliken M, Saporta S, Zigova T, Cahill T, et al. Positive effect of transplantation of hNT neurons (NTera 2/D1 cell line) in a model of familiar amyotrophic lateral sclerosis. Exp Neurol 2002; 174: 169-180.
42. Garbuzova-Davis S, Willing AE, Zigova T, Saporta S, Justen EB, Lane JC, et al. Intravenous administration of human umbilical cord blood cells in a mouse model of amyotrophic lateral sclerosis: distribution, migration, and differentiation. J Hematother Stem Cell Res 2003; 12: 255-270.
43. Corti S, Locatelli F, Papadimitriou D, Del Bo R, Nizzardo M, Nardini M, et al. Neural stem cells Lewis X+ CXCR4+ modify disease progression in an amyotrophic lateral sclerosis model. Brain 2007; 130: 1289-1305.
44. Zhao C P, Zang C, Wang YH, Zhou SN, Zhou C, Li WY, et al. Signals in pathological CNS extracts of ALS mice promote hMSC neurogenic differentiation in vitro. Cell Biol Int 2007; 31: 1428-1435.
45. Wada R, Tift CJ, Proia RL. Microglial activation precedes acute neurodegeneration in Sandhof disease and is suppressed by bone marrow transplantation. Proc Natl Acad Sci U S A 2000; 97: 10954-10959.
46. Wu Y P, McMahon EJ, Matsuda J, Suzuki K, Matsushima GK, Suzuki K. Expression of immune-related molecules is downregulated in twitcher mice following bone marrow transplantation. J Neuropathol Exp Neurol 2001; 60: 1062-1074.
47. Priller J, Flügel A, Wehner T, Boentert M, Haas CA, Prinz M, et al. Targeting gene modifed hematopoietic cells to the central nervous system: use of green fuorescent protein uncovers microglial engraftment. Nat Med 2001; 7: 1356-1361.
48. Bif A, De Palma M, Quattrini A, Del Carro U, Amadio S, Visigalli I, et al. Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modifed hematopoietic stem cells. J Clin Invest 2004; 113: 1118-1129.
49. Neumann H. Microglia: a cellular vehicle for CNS gene therapy. J Clin Invest 2006; 116: 2857-2860.
50. Pluchino S, Quattrini A, Brambilla E, Gritti A, Salani G, Dina G, et al. Injection of adult neurospheres induces recovery in a chronic model of multiple sclerosis. Nature 2003; 422: 688-694.
51. Janson CG, Ramesh TM, During MJ, Leone P, Heywood J. Human intrathecal transplantation of peripheral blood stem cells in amyotrophic lateral sclerosis. J Hematother Stem Cell Res 2001; 10: 913-915.
52. Van den Berg DJ, Sharma AK, Bruno E, Hofman R. Role of members of the Wnt gene family in human hematopoiesis. Blood 1998; 92: 3189-3202.
53. Le Blanc K, Tammik L, Sundberg B, Haynesworth SE, Ringdén O. Mesenchymal stem cells inhibit and stimulate mixed lymphocyte cultures and mitogenic responses independently of the major histocompatibility complex. Scand J Immunol 2003; 57: 11-20.
54. Nauta A, Fibbe W. Immunomodulatory properties of mesenchymal stromal cells. Blood 2007; 110: 3499-3506.
55. Ortiz LA, Dutreil M, Fattman C, Pandey AC, Torres G, Go K, et al. Interleukin 1 receptor antagonist mediates the antiinfammatory and antifbrotic effect of mesenchymal stem cells during lung injury. Proc Natl Acad Sci U S A 2007; 104: 11002-11007.
56. Di Nicola M, Carlo-Stella C, Magni M, Milanesi M, Longoni PD, Matteucci P, et al. Human bone marrow stromal cells suppress T-lymphocyte proliferation induced by cellular or nonspecifc mitogenic stimuli. Blood 2002; 99: 3838-3843.
57. Chiang KY, Lamb L, Clark J, Worthington-White D, Rich I, Henslee-Downey PJ. Assessment of G-CSF stimulated BM hematopoietic stem cells in normal donors. Cytotherapy 2002; 4: 55-63.
58. Meisel R, Zibert A, Laryea M, Göbel U, Däubener W, Dilloo D. Human bone marrow stromal cells inhibit allogeneic T-cell responses by indoleamine 2,3-dioxygenase-mediated tryptophan degradation. Blood 2004; 103: 4619-4621.
59. Sato K, Ozaki K, Oh I, Meguro A, Hatanaka K, Nagai T, et al. Nitric oxide plays a critical role in suppression of T-cell proliferation by mesenchymal stem cells. Blood 2007; 109: 228-234.
60. Nasef A, Mathieu N, Chapel A, Frick J, François S, Mazurier C, et al. Immunosuppressive effect s of mesenchymal stem cells: involvement of HLA-G. Transplantation 2007; 84: 231-237.
61. Abdi R, Fiorina P, Adra CN, Atkinson M, Sayegh MH. Immunomodulation by mesenchymal stem cells: a potential therapeutic strategy for type 1 diabetes. Diabetes 2008; 57: 1759-1767.
62. Uccelli A, Zappia E, Benvenuto F, Frassoni F, Mancardi G. Stem cells in infammatory demyelinating disorders: a dual role for immunosuppression and neuroprotection. Expert Opin Biol 2006; 6: 17-22.
63. Rice CM, Scolding NJ. Adult stem cells -reprogramming neurological repair? Lancet 2004; 364: 193-199.
64. Barkho BZ, Song H, Aimone JB, Smart RD, Kuwabara T, Nakashima K, et al. Identifcation of astrocyte-expressed factors that modulate neural stem/progenitor cell differentiation. Stem Cells Dev 2006; 15: 407-421.
65. Turner MR, Parton MJ, Leigh PN. Clinical trials in ALS: an overview. Semin Neurol 2001; 21: 167-175. Clinical Trials. URL: http://clinicaltrials.gov/. [15.07.2010].
66. Mitchell JD, Borasio GD. Amyotrophic lateral sclerosis. Lancet 2007; 369: 2031-2041.
67. Brooks BR, Miller RG, Swash M, Munsat TL; World Federation of Neurology Research Group on Motor Neuron Diseases. El Escorial revised criteria for the diagnosis of amyotrophic lateral sclerosis. Amyotroph Lateral Scler Other Motor Neuron Disord 2000; 1: 293-299.
68. Mazzini L, Mareschi K, Ferrero I, Vassallo E, Oliveri G, Nasuelli N, et al. Stem cell treatment in amyotrophic lateral sclerosis. J Neurol Sci 2008; 265: 78-83.
69. Te ALS-CNTF Treatment Study (ACTS) Phase I-II Study Group. Te Amyotrophic Lateral Sclerosis Functional Rating Scale: assessment of activities of daily living in patients with amyotrophic lateral sclerosis. Arch Neurol 1996; 53: 141-147.
70. Norris FH, Calanchini PR, Fallat RJ, Panchari S, Jewett B. The administration of guanidine in amyotrophic lateral sclerosis. Neurology 1974; 24: 721-728.
71. Mazzini L, Vercelli A, Mareschi K, Ferrero I, Testa L, Fagioli F. Mesenchymal stem cells for ALS patients. Amyotroph Lateral Scler 2009; 10: 123-124.
72. Appel SH, Engelhardt JI, Henkel JS, Siklos L, Beers DR, Yen AA, et al. Hematopoietic stem cell transplantation in patients with sporadic amyotrophic lateral sclerosis. Neurology 2008; 71: 1326-1334.
73. Haverkamp L, Appel V, Appel SH. Natural history of amyotrophic lateral sclerosis in a database population: validation of a scoring system and a model for survival prediction. Brain 1995; 118: 707-792.
74. Cashman N, Tan LY, Krieger C, Mädler B, Mackay A, Mackenzie I, et al. Pilot study of granulocyte colony stimulating factor (G-CSF)-mobilized peripheral blood stem cells in amyotrophic lateral sclerosis (ALS). Muscle Nerve 2008; 37: 620-625.
75. Deda H, Inci MC, Kürekçi AE, Sav A, Kayihan K, Özgün E, et al. Treatment of amyotrophic lateral sclerosis patients by autologous bone marrow-derived hematopoietic stem cell transplantation: a 1-year follow-up. Cytotherapy 2009; 11: 16-25.
76. Martínez HR, González-Garza MT, Moreno-Cuevas JE, Caro E, Gutiérrez-Jiménez E, Segura JJ. Stem-cell transplantation into the frontal motor cortex in amyotrophic lateral sclerosis patients. Cytotherapy 2009; 11: 26-34.
77. Silani V, Cova L, Corbo M, Ciammola A, Polli E. Stem cell therapy for amyotrophic lateral sclerosis. Lancet 2004; 364: 200-222.
78. Samijn JP, Te Boekhorst PA, Mondria T, Van Doorn PA, Flach HZ, Van der Meché FG, et al. Intense T cell depletion followed by autologous bone marrow transplantation for severe multiple sclerosis. J Neurol Neurosurg Psychiatry 2006; 77: 46-50.